National Research Centre

Medical Research Ethics Committee

MREC Reviewer Comment

Protocol Title

Real-World Effectiveness and Safety of Vosoritide in Children with Achondroplasia: A Multicenter Retrospective Study in the Middle East

Completely satisfied and informative data

Needs Explanation

Comments to the applicant

-Fund: Self or in collaboration with the listed funding agent? It is mentioned that the protocol will be developed by the investigators in collaboration with BioMarin Pharmaceutical and Connect Communications. Connect Communications will support with the data collation, data analysis, and manuscript development.

 

-In the cover letter it is written that it is expected to have 80-120 participating patients, while in the proposal it is written that only 15 will be participating; which number is correct?

- In the cover letter it is written that different countries in the middle east region are included in the study, but in the proposal only Center of Excellence of Medical Research, National Research Centre, and The Department of Pediatrics, Faculty of Medicine, Menoufia University are mentioned.

نموذج 90 غير موضح به إذا كان البروتولول سليم علميا و تمت الموافقة عليه أم لا

 

Comment to the Committee

Informed consent form:

Waiver

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